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日前，启明创投投资企业信念医药宣布，信玖凝®（BBM-H901注射液，通用名称：波哌达可基注射液）已正式获得中国国家药品监督管理局（NMPA）批准，用于中重度血友病B（先天性凝血因子IX缺乏症）成年患者的治疗。信玖凝®是中国首个获批的血友病B基因治疗药物，同时也是中国首个获批的重组腺相关病毒（rAAV）基因治疗药物。

信念医药联合创始人、董事长兼首席科学家肖啸博士表示：“信念医药始终致力于前沿基因治疗领域的研发和创新，以患者需求为核心，突破技术壁垒，致力于填补未被满足的临床需求。此次获批是信念医药发展历程中的重要里程碑，作为中国自主研发的首款血友病B基因治疗药物，信玖凝®将为血友病B患者提供一种全新的治疗方案，有望帮助患者摆脱需要终生频繁静脉注射用药的困扰，回归自由人生。未来，信念医药将努力推进该项研究成果的海外商业化进程，期望能够惠及更多血友病B患者。”

信念医药联合创始人兼首席执行官郑静博士表示：“信玖凝®从研发到上市用了不到七年的时间，这种快速、高效的成果转化有赖于信念人的专业专注和不懈努力，及行业各界的精诚合作和有力支持。信玖凝®作为中国首个获批的重组腺相关病毒（rAAV）基因治疗药物，预示着中国基因治疗行业迈入了新的里程碑，也凸显了信念医药在基因治疗领域的突破性优势。依托先进的腺相关病毒（AAV）衣壳发现平台Capsidx®和数百种载体关键技术及自主商业化生产平台，信念医药已建立起了丰富的研发管线，治疗领域覆盖罕见遗传疾病，如血友病、杜氏肌营养不良，及慢性疾病如骨关节炎病、帕金森、宫颈内瘤上皮样病变等多种未被满足临床需求的疾病。未来，信念医药将持续深耕基因治疗领域，让尖端科技惠及更多生命，实现服务全球患者的愿景。” 

2022年，信玖凝®由研究者发起的研究（IIT）成果先后发表于国际权威期刊《柳叶刀-血液病学》和《新英格兰医学杂志》。2024年，信玖凝® IIT 3年以上长期随访结果在2024国际血栓与止血学会（ISTH）大会上以口头报告形式展示。同年，信念医药在第66届美国血液学会（ASH）年会正式发布信玖凝®3期临床研究结果。研究结果显示，相较于预防性FIX替代疗法，信玖凝®能够显著降低患者的年化出血率（ABR），快速提升患者体内FIX活性水平并持续稳定表达，具有良好的安全性。以3期临床研究结果为例，接受基因治疗后随访52周时，受试者年化出血率（ABR）均值为0.6，平均FⅨ活性达到55.08 IU/dL（基于SynthaSIL aPTT试剂的一期法），外源性FⅨ药物的平均输注次数从治疗前的58.2次/年降至治疗后的2.9次/年。26名受试者中的21位（80.8%）在治疗后未出现出血事件，且所有受试者均无严重不良事件发生。

2022年8月被中国国家药品监督管理局药品审评中心纳入“突破性治疗品种”名单，同月获得美国食品药品监督管理局（FDA）的孤儿药认定（ODD）；2024年获得FDA儿科罕见病资格认定（RPDD）以及欧洲药品管理局（EMA）先进治疗药物分类认证（ATMP）。

信念医药此前与武田中国达成独家合作协议，由信念医药研发生产信玖凝®，武田中国负责该产品在中国内地、中国香港和中国澳门的商业化进程。双方整合了各自资源优势，加速为患者提供突破性的基因疗法，共同开拓血友病B治疗领域新格局。

Belief BioMed announced BBM-H901 (Dalnacogene Ponparvovec Injection), China's first hemophilia B gene therapy, was officially approved

Belief BioMed, a portfolio company of Qiming Venture Partners, announced that BBM-H901 (generic name: Dalnacogene Ponparvovec Injection), has been officially approved by China's National Medical Products Administration (NMPA) for the treatment of adult patients with moderate to severe hemophilia B (congenital coagulation factor IX deficiency). BBM-H901 is the first approved hemophilia B gene therapy in China, and is also the first approved gene therapy in China based on recombinant adeno-associated virus (rAAV) vector.

Dr. Xiao Xiao, Co-founder, Chairman and Chief Science Officer of Belief BioMed, said: "Belief BioMed has always been committed to R&D and innovation in the field of cutting-edge gene therapy, and is dedicated to fulfilling the unmet medical needs. The approval of BBM-H901 is an important milestone along the development journey of Belief BioMed. As the first hemophilia B gene therapy drug independently developed in China, BBM-H901 will provide a new treatment option for hemophilia B patients, which is expected to provide patients with an innovative treatment solution and help them return to a normal life. In the future, Belief BioMed will strive to accelerate the overseas commercialization process of BBM-H901, hoping to benefit more hemophilia B patients."

Dr. Jane Zheng, Co-founder and Chief Executive Officer of Belief BioMed, said: "The market approval of BBM-H901 takes less than seven years from R&D to launch, which is a remarkable rapid speed. This is not only generated from the dedication and unremitting efforts of our employees, but also from the strong supports given by our external partners. The approval of BBM-H901, the first approved recombinant adeno-associated virus (rAAV) gene therapy in China, marks a new era of China's gene therapy industry and highlights Belief BioMed's advantages in the field. Relying on the advanced AAV capsid discovery platform Capsidx®, hundreds of key vector technologies, as well as an independent production platform, Belief BioMed has established an extensive R&D pipeline, including the pipelines on rare genetic diseases such as hemophilia and Duchenne muscular dystrophy, and those on chronic diseases such as osteoarthritis, Parkinson's disease and cervical intraepithelial neoplasia. All of those are developed to fulfill the high levels of unmet medical needs. In the future, Belief BioMed would continue to focus on gene therapy to bring cutting-edge technology to more patients and achieve our vision of serving patients worldwide."  

BBM-H901, based on a recombinant adeno-associated virus (rAAV) vector, can deliver the optimized human coagulation FIX gene into liver cells of patients. Then, coagulation FIX is continuously expressed and secreted into the bloodstream using the host cell gene transcription system, thereby promoting coagulation.

In 2022, the research results of first Investigator-initiated Trial (IIT) of BBM-H901 were successively published on international authoritative journals, The Lancet-Hematology and The New England Journal of Medicine. In 2024, the IIT long-term follow-up results of more than 3 years were orally presented as an oral presentation at the 2024 International Society on Thrombosis and Haemostasis (ISTH) Congress, and in the same year, Belief BioMed released the results of the Phase 3 clinical study at the 66th Annual Meeting of the American Society of Hematology (ASH). 

The current results showed that compared to treatment efficacy of preventive factor IX (FIX) replacement therapy, the annualized bleeding rate (ABR) of the patient was significantly reduced, and the FIX activity was rapidly increased and maintained stable expression, while showing good safety. 

As an example, the results of BBM-H901 Phase 3 clinical study showed that after 52 weeks of follow-up, the mean annualized bleeding rate (ABR) of the participants was 0.6, the average FIX activity reached 55.08 IU/dL (one Stage Assay  SynthaSIL aPTT reagent), and the average number of infusions of FIX drug decreased from 58.2 times/year before gene therapy treatment to 2.9 times/year after that. Twenty-one of the 26 participants (80.8%) had no bleeding events after treatment, and all participants had no SAEs.

In August 2022, BBM-H901 obtained the Breakthrough Therapy Designation by the Center for Drug Evaluation, China's National Medical Products Administration, and later on Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). In 2024, BBM-H901 obtained the Rare Pediatric Disease Designation (RPDD) from the FDA and the Advanced Therapy Medical Products (ATMP) from the European Medicines Agency (EMA).

Belief BioMed signed an exclusive cooperation agreement with Takeda China. According to the agreement, Belief BioMed granted Takeda China the exclusive license for the commercialization of BBM-H901 in Chinese Mainland, Hong Kong, and Macau.